{"id":"W1569820390","doi":"10.1002/jgm.2768","title":"Adeno‐associated virus 8‐mediated gene therapy for choroideremia: preclinical studies in <i>in vitro</i> and <i>in vivo</i> models","year":2014,"lang":"en","type":"article","venue":"The Journal of Gene Medicine","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":31,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Foundation Fighting Blindness; Pennsylvania Department of Health; University of Pennsylvania; Research to Prevent Blindness; F. M. Kirby Foundation; Choroideremia Research Foundation","keywords":"Choroideremia; In vivo; Adeno-associated virus; Biology; Genetic enhancement; In vitro; Retinal; Retinal pigment epithelium; Rab; Retinal degeneration; Gene delivery; Molecular biology; Western blot; Virology; Cell biology; Recombinant DNA; Gene; Vector (molecular biology); Biochemistry; Genetics","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.002595615,0.0001562971,0.0004410182,0.0001026111,0.00003221711,0.000003816119,0.0001896048,0.0001261536,0.000002696829],"category_scores_gemma":[0.0007535794,0.00009572644,0.00004846018,0.000141107,0.0001704167,0.000007512049,0.00003579253,0.0001919488,2.066477e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002827336,"about_ca_system_score_gemma":0.0000641625,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00002803576,"about_ca_topic_score_gemma":0.0001934638,"domain_scores_codex":[0.9985399,0.0002299521,0.0006561999,0.000153967,0.0001854007,0.0002346284],"domain_scores_gemma":[0.9991373,0.0002949484,0.0002667374,0.0001230793,0.0001178415,0.00006004376],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.003085799,0.0001152043,0.003293431,0.00001488679,0.00008893078,0.000005674217,0.0008554707,0.0003668985,0.9888251,0.000002019881,0.001903682,0.001442909],"study_design_scores_gemma":[0.04335231,0.005092605,0.02206615,0.0005633305,0.0001531593,0.0001437491,0.002496412,0.003116375,0.9098033,0.005524676,0.006958779,0.0007291814],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.971303,0.02589638,0.000355516,0.001941987,0.0001507128,0.0002442651,0.000003755307,0.000001978711,0.0001023339],"genre_scores_gemma":[0.9610021,0.0372109,0.0002484641,0.001227969,0.0001991799,0.00000957308,0.00001194584,0.00001605779,0.00007380473],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.07902183,"threshold_uncertainty_score":0.3903611,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04159326182806776,"score_gpt":0.3213658012579557,"score_spread":0.2797725394298879,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}